How to Rebuild Trust Within the Sickle Cell Community For Clinical Trials After a Drug Failure Like Pfizer’s Voxelotor
People with sickle cell disease have to navigate many ups and downs when it comes to their health. Stress or something as simple as a change in the weather can trigger a painful crisis.
Longstanding treatment options and newer therapies can help sickle cell warriors combat the pain and recover from a crisis. There are exciting new developments from pharmaceutical companies, but with progress comes challenges and even setbacks.
For example, Pfizer voluntarily withdrew the drug Oxbryta or voxelotor in 2024 over safety concerns after ongoing studies found a higher rate of severe pain crises and increased deaths.
In the Sickle Cell Foundation of Georgia’s new TV series “Unveiling Sickle Cell: Beyond the Pain,” host and foundation CEO Tabatha McGee explored how this type of drug failure can impact clinical trials and the trust of the sickle cell community.
Because of the historical mistrust of the African American community in traditional western medicine in the United States, it can be difficult to put together clinical trials for new sickle cell drugs.
“But we’ve been very fortunate to have had good partnerships with some of the pharmaceutical companies and helping to build that trust, and many people here in Atlanta were subjects in trials that have led to FDA-approved drugs,” longtime sickle cell disease (SCD)and hematology researcher Dr. Beatrice Gee told McGee during an interview on “Unveiling Sickle Cell.”
Gee is the Theresa Wright Chair of Sickle Cell Disease Research and the director of the Aflac Sickle Cell Disease Program at Children’s Healthcare of Atlanta. She’s also an acting professor of pediatrics at Emory University School of Medicine and an adjunct professor of pediatrics at Morehouse School of Medicine.
Gee acknowledged that the withdrawal of voxelotor has caused concern in the sickle cell community, and she noted that clinical trials are essential to developing new treatments and drugs. She told McGee it’s going to take some time to figure out how the drug withdrawal will impact clinical trial recruitment.
“The community is very disappointed in the failure,” McGee told Gee and her other guest Eldrida Randal, a clinical researcher and transition coordinator who has more than 35 years of experience at Emory University and three decades of experience at Grady Health System as a clinical researcher and transition coordinator.
“That new finding and the halting of that drug has impacted our patients in a way that’s heart wrenching for me, as well, because we’ve worked so hard as research coordinators helping, you know, trying to build trust and trying to assure them that we’re out there for their best interests,” Randal told McGee.
“How do we rebuild that trust? And one of the first things I think we need to do is have a very open conversation and be honest with the patients,” Randal added.
McGee explored the development of gene therapies with Gee and Randal. The FDA approved two gene therapies in 2023 for people 12 and older with SCD.
“Gene therapy getting approved by the FDA is a great milestone for people with sickle cell disease because we’ve been talking about this process of being able to fix the sickle cell mutation for decades,” Gee told McGee.
“So, there are two products that are FDA-approved and what they do is they change the genes within the blood-forming cells of the person with sickle cell disease,” Gee explained.
Both treatments, Casgevy and Lyfgenia, are intended to be a lifetime fix, but they work in different ways and require years of foll-up research to make sure
“There’s a mutation that causes the beta hemoglobin to have a sickle cell mutation and what these therapies do is either they cut the mutation out or they give a new gene that helps counteract the sickle cell mutation so that the cells will then make new red blood cells that either don’t have the sickle cell mutation or just don’t sickle as much,” Gee added.
But these treatments are so expensive, $2 to $3 million per person with additional hospital costs on top of that. So right now, these treatments are not viable for the average American with sickle cell disease. It’s also not covered by insurance companies right now.
Of those who have had the treatment, it has helped some sickle cell warriors.
“It’s a very hopeful treatment, but right now it’s awfully expensive,” Gee said.
-By Shelby Lin Erdman